The Indian Institute of Technology Jodhpur has established a Research Centre in collaboration with Dystrophy Annihilation Research Trust (DART) Bangalore and the All India Institute of Medical Sciences (AIIMS) Jodhpur to research about Duchenne Muscular Dystrophy (DMD), a rare genetic disease
Many challenges are faced concerning DMD, which is a rare and incurable genetic disease. DMD is the most common and fatal type of muscular dystrophy, marked by gradual muscle deterioration. The condition is predominantly seen in boys.
In India, over five lakh boys are suffering from DMD. The centre at IIT Jodhpur aims to develop affordable therapeutics for DMD and enhance the efficacy of Antisense Oligonucleotide (AON)-based therapeutics.
The AON-based therapeutics’ idea is to hide or mask specific exons (a segment of a DNA or RNA molecule containing information coding for a protein) in a gene sequence. In DMD patients, one or more exons can be masked with specific molecules called AON or molecular patches.
Due to these challenges, DMD patients need personalized medicine. The current therapeutic options available to treat DMD are minimal and highly expensive (over Rs. 2-3 crores per child a year). Drugs are mostly imported from abroad, accelerating dosing costs and putting them out of reach for most families.
The main focus areas of the DMD Research Centre at IIT Jodhpur is to design an affordable and effective therapeutic for DMD patients by adopting a bioengineered concept under the “Make in India” (AtmaNirbhar) initiative.
“Another focus is to conduct social outreach programmes to generate awareness about this condition and extend support for children with DMD and their parents,” stated the press statement issued.
Talking about the DMD Research Centre at IIT Jodhpur, Prof. Surajit Ghosh, Dean Research and Development, Professor of Bioscience & Bioengineering, IIT Jodhpur, said, “The primary goal of our team is to develop two therapeutic leads for clinical trials on high priority”.